Shanghai Zhimeng Biopharma has completed patient enrolment in the pivotal Phase II/III clinical trial of its investigational potassium-channel opener, CB03-154, for amyotrophic lateral sclerosis (ALS).
A total of 255 patients have been recruited across 15 clinical research centres in mainland China, and Professor Fan Dongsheng of the 3rd Affiliated Hospital of Peking University is the principal investigator.
The trial’s primary efficacy endpoint is the change from baseline in Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score at week 39.
Discovered and developed by Zhimeng Biopharma independently, CB03-154 showed neuroprotective effects on motor neurons through modulation of neuronal excitability, restoring the balance between excitation and inhibition, in preclinical studies.
Zhimeng Biopharma founder and CEO Dr Huanming Chen said: “I would like to extend my sincere appreciation to Professor Fan Dongsheng and the investigators at all clinical research centres across China for their dedicated efforts, as well as to all ALS patients and their families who participated in the trial for their selfless commitment.
“Moving forward, we will spare no effort to advance clinical development and regulatory submissions, striving to bring a truly effective new treatment option to ALS patients in China and around the world as soon as possible.”
The China Centre for Drug Evaluation (CDE) approved the initiation of the Phase II/III ALS trial in July 2025. The US Food and Drug Administration had previously granted CB03-154 orphan drug designation in October 2023.
If Phase II top-line data prove positive in the third quarter of 2027, the company intends to file a marketing application with the China CDE seeking conditional approval.
Zhimeng Biopharma said this approach is designed to give ALS patients earlier access to innovative treatments, reflecting its patient-centred development philosophy in rare diseases.
Beyond ALS, CB03-154 is advancing through clinical development in additional central nervous system (CNS) indications. As of October 2026, the drug had entered Phase II trials for focal epilepsy in both Australia and China.
